A note on sample size calculations for cluster randomised crossover trials with a fixed number of clusters.

abstract：:Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...

journal_title：Statistics in medicine

authors： Temple R

更新日期：2002-10-15 00:00:00

abstract：:The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful t...

journal_title：Statistics in medicine

authors： Ukoumunne OC,Davison AC,Gulliford MC,Chinn S

更新日期：2003-12-30 00:00:00

abstract：:In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

journal_title：Statistics in medicine

authors： Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

更新日期：2019-11-30 00:00:00

abstract：:For many HIV-infected patients, use of antiretroviral therapy (ART) results in a sustained suppression of plasma viral load to undetectable levels. However, due to lack of antigenic stimulation, this may also result in a gradual loss of cell-mediated immune (CMI) responses that help control HIV infection. In concept, ...

journal_title：Statistics in medicine

authors： Mogg R,Mehrotra DV

更新日期：2007-02-10 00:00:00

abstract：:Risk prediction models play an important role in prevention and treatment of several diseases. Models that are in clinical use are often refined and improved. In many instances, the most efficient way to improve a successful model is to identify subgroups for which there is a specific biological rationale for improvem...

journal_title：Statistics in medicine

authors： Biswas S,Arun B,Parmigiani G

更新日期：2014-05-20 00:00:00

abstract：:Individuals may vary in their responses to treatment, and identification of subgroups differentially affected by a treatment is an important issue in medical research. The risk of misleading subgroup analyses has become well known, and some exploratory analyses can be helpful in clarifying how covariates potentially i...

journal_title：Statistics in medicine

authors： Kim HJ,Lu B,Nehus EJ,Kim MO

更新日期：2019-02-10 00:00:00

abstract：:The lifetime risk of a clinical condition is the probability of onset of the condition during one's lifespan. Recent advances in Alzheimer's disease (AD) research have identified screening tests for biomarkers that can identify persons who are in the earliest stages of the AD process but who do not yet have any clinic...

journal_title：Statistics in medicine

authors： Brookmeyer R,Abdalla N

更新日期：2019-04-30 00:00:00

abstract：:Obtaining accurate estimates of the performance of a diagnostic test for some population of patients might be difficult when the sample of subjects used for this purpose is not representative for the whole population. Thus, in the motivating example of this paper a test is evaluated by comparing its results with those...

journal_title：Statistics in medicine

authors： Buzoianu M,Kadane JB

更新日期：2008-06-15 00:00:00

abstract：:The Jimma Infant Survival Differential Longitudinal Study is an Ethiopian study, set up to establish risk factors affecting infant survival and to investigate socio-economic, maternal and infant-rearing factors that contribute most to the child's early survival. Here, a subgroup of about 1500 children born in Jimma to...

journal_title：Statistics in medicine

authors： Lesaffre E,Asefa M,Verbeke G

更新日期：1999-04-15 00:00:00

abstract：:I reflect on the statistical methods of the Christakis-Fowler studies on network-based contagion of traits by checking the sensitivity of these kinds of results to various alternate specifications and generative mechanisms. Despite the honest efforts of all involved, I remain pessimistic about establishing whether bin...

journal_title：Statistics in medicine

authors： Thomas AC

更新日期：2013-02-20 00:00:00

abstract：:Binary classification rules based on covariates typically depend on simple loss functions such as zero-one misclassification. Some cases may require more complex loss functions. For example, individual-level monitoring of HIV-infected individuals on antiretroviral therapy requires periodic assessment of treatment fail...

journal_title：Statistics in medicine

authors： Xu Y,Liu T,Daniels MJ,Kantor R,Mwangi A,Hogan JW

更新日期：2019-05-20 00:00:00

abstract：:Biomarkers that predict the efficacy of treatment can potentially improve clinical outcomes and decrease medical costs by allowing treatment to be provided only to those most likely to benefit. We consider the design of a randomized clinical trial in which one objective is to evaluate a treatment selection marker. The...

journal_title：Statistics in medicine

authors： Janes H,Brown MD,Pepe MS

更新日期：2015-11-30 00:00:00

abstract：:The identification of counties burdened by exceptionally high rates of mortality is a fundamental step in the development of state-based intervention and prevention strategies. However, the estimation of rates from small geographic areas presents special problems, especially for rare events. This paper compares the us...

journal_title：Statistics in medicine

authors： Stevenson JM,Olson DR

更新日期：1993-02-01 00:00:00

abstract：:We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...

journal_title：Statistics in medicine

authors： Zhou SY,Kingsley LA,Taylor JM,Chmiel JS,He DY,Hoover DR

更新日期：1993-01-30 00:00:00

abstract：:Instrumental variable is an essential tool for addressing unmeasured confounding in observational studies. Two-stage predictor substitution (2SPS) estimator and two-stage residual inclusion (2SRI) are two commonly used approaches in applying instrumental variables. Recently, 2SPS was studied under the additive hazards...

journal_title：Statistics in medicine

authors： Ying A,Xu R,Murphy J

更新日期：2019-05-10 00:00:00

abstract：:Comparative Poisson trials of prophylactic interventions, such as vaccines, can be lengthy and costly. We evaluate two easily implemented approaches to reduce numbers of disease cases and person years of follow up (N(u+t)) for comparative Poisson trials with fixed numbers of cases (T); (i) altering k the portion of N(...

journal_title：Statistics in medicine

authors： Hoover DR

更新日期：2004-04-30 00:00:00

abstract：:Many studies have reported large variations in age- and sex-adjusted rates of hospitalizations across small geographic areas. These variations have often been attributed to differences in medical practice style which are not reflected in differences in health care outcomes. There is, however, another potentially impor...

journal_title：Statistics in medicine

authors： Peköz EA,Shwartz M,Iezzoni LI,Ash AS,Posner MA,Restuccia JD

更新日期：2003-05-30 00:00:00

abstract：:This paper considers the regression analysis of CD4 cell counts, a commonly used indicator and prognostic factor of AIDS progression. For this purpose, a number of methods have been proposed and most of them are based on random effects models. We present an alternative that is based on a mean function regression model...

journal_title：Statistics in medicine

authors： Sun J

更新日期：2001-11-15 00:00:00

abstract：:Data monitoring committees (DMCs) have become an increasingly common component of randomized clinical trials in recent years. As experience has accumulated, and more individuals and organizations have become involved in such activities, a variety of approaches to the operation of such committees has inevitably arisen....

journal_title：Statistics in medicine

authors： Ellenberg SS

更新日期：2001-09-15 00:00:00

abstract：:Timeliness of a public health surveillance system is one of its most important characteristics. The process of predicting the present situation using available incomplete information from surveillance systems has received the term nowcasting and has high public health interest. Generally in Europe, general practitione...

journal_title：Statistics in medicine

authors： Nunes B,Natário I,Lucília Carvalho M

更新日期：2013-07-10 00:00:00

abstract：:Blinded estimation of variance allows for changing the sample size without compromising the integrity of the trial. Some of the methods that estimate the variance in a blinded manner either make untenable assumptions or are only applicable to two-treatment trials. We propose a new method for continuous endpoints that ...

journal_title：Statistics in medicine

authors： Xing B,Ganju J

更新日期：2005-06-30 00:00:00

abstract：:We extend the method proposed in a recent work by the Authors for trial-level general surrogate evaluation to allow combinations of biomarkers and provide a procedure for finding the "best" combination of biomarkers based on the absolute prediction error summary of surrogate quality. We use a nonparametric Bayesian mo...

journal_title：Statistics in medicine

authors： Gabriel EE,Sachs MC,Daniels MJ,Halloran ME

更新日期：2019-03-30 00:00:00

abstract：:In an individually matched case-control study, effects of potential risk factors are ascertained through conditional logistic regression (CLR). Extension of CLR to situations with multiple disease or reference categories has been made through polychotomous CLR and is shown to be more efficient than carrying out separa...

journal_title：Statistics in medicine

authors： Mukherjee B,Liu I,Sinha S

更新日期：2007-07-30 00:00:00

abstract：:We consider the use of the assurance method in clinical trial planning. In the assurance method, which is an alternative to a power calculation, we calculate the probability of a clinical trial resulting in a successful outcome, via eliciting a prior probability distribution about the relevant treatment effect. This i...

journal_title：Statistics in medicine

authors： Ren S,Oakley JE

更新日期：2014-01-15 00:00:00

abstract：:Equivalence trials aim to show that two treatments have equivalent therapeutic effects. The approach is to define, in advance, a range of equivalence -d to +d for the treatment difference such that any value in the range is clinically unimportant. If the confidence interval for the difference, calculated after the tri...

journal_title：Statistics in medicine

authors： Ebbutt AF,Frith L

更新日期：1998-08-15 00:00:00

abstract：:The FDA permits marketing of a generic formulation of a drug G for the same indications as a standard preparation S if one can show that G is bioequivalent to S. Present implementation requires convincing evidence that the population mean difference in bioavailability (drug exposure) between the two preparations lies ...

journal_title：Statistics in medicine

authors： Sheiner LB

更新日期：1992-09-30 00:00:00

abstract：:This paper discusses regression analysis of multivariate current status failure time data (The Statistical Analysis of Interval-censoring Failure Time Data. Springer: New York, 2006), which occur quite often in, for example, tumorigenicity experiments and epidemiologic investigations of the natural history of a diseas...

journal_title：Statistics in medicine

authors： Chen MH,Tong X,Sun J

更新日期：2009-11-30 00:00:00

abstract：:A popular method to control multiplicity in confirmatory clinical trials is to use a so-called hierarchical, or fixed sequence, test procedure. This requires that the null hypotheses are ordered a priori, for example, in order of clinical importance. The procedure tests the hypotheses in this order using alpha-level t...

journal_title：Statistics in medicine

authors： Edwards D,Madsen J

更新日期：2007-12-10 00:00:00

abstract：:The continual reassessment method (CRM) is an adaptive design for Phase I trials whose operating characteristics, including appropriate sample size, probability of correctly identifying the maximum tolerated dose, and the expected proportion of participants assigned to each dose, can only be determined via simulation....

journal_title：Statistics in medicine

authors： Braun TM

更新日期：2020-09-16 00:00:00

abstract：:We consider the analysis of serial biomarkers to screen and monitor individuals in a given population for onset of a specific disease of interest. The biomarker readings are subject to error. We survey some of the existing literature and concentrate on two recently proposed models. The first is a fully Bayesian hierar...

journal_title：Statistics in medicine

authors： Slate EH,Turnbull BW

更新日期：2000-02-29 00:00:00